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2021年血友病治疗:采用综合、以患者为导向的方法,在患者和临床医生之间进行共同决策,选择“最佳”治疗方案。

Hemophilia treatment in 2021: Choosing the"optimal" treatment using an integrative, patient-oriented approach to shared decision-making between patients and clinicians.

作者信息

Hermans Cedric, Noone Declan, Benson Gary, Dolan Gerry, Eichler Hermann, Jiménez-Yuste Víctor, Königs Christoph, Lobet Sébastien, Pollard Debra, Zupančić-Šalek Silva, Mancuso Maria Elisa

机构信息

Haemostasis and Thrombosis Unit, Division of Haematology, Cliniques Universitaires Saint-Luc, Université catholique de Louvain (UCLouvain), Brussels, Belgium.

Irish Haemophilia Society, Dublin, Ireland.

出版信息

Blood Rev. 2022 Mar;52:100890. doi: 10.1016/j.blre.2021.100890. Epub 2021 Sep 29.

Abstract

The mainstay of hemophilia treatment is to prevent bleeding through regular long-term prophylaxis and to control acute breakthrough bleeds. Various treatment options are currently available for prophylaxis, and treatment decision-making is a challenging and multifaceted process of identifying the most appropriate option for each patient. A multidisciplinary expert panel convened to develop a practical, patient-oriented algorithm to facilitate shared treatment decision-making between clinicians and patients. Key variables were identified, and an algorithm proposed based on five variables: bleeding phenotype, musculoskeletal status, treatment adherence, venous access, and lifestyle. A complementary, patient-focused preference tool was also hypothesized, with the aim of exploring individual patients' priorities, preferences, and goals. It is hoped that the proposed algorithm and the hypothesized patient preference tool will assist in selecting a treatment for each patient that is as efficient as possible in preventing bleeds while also accounting for the patient's expectations and priorities.

摘要

血友病治疗的主要方法是通过定期长期预防来防止出血,并控制急性突破性出血。目前有多种预防治疗方案可供选择,而治疗决策是一个具有挑战性且多方面的过程,需要为每位患者确定最合适的方案。一个多学科专家小组召开会议,制定了一个实用的、以患者为导向的算法,以促进临床医生和患者之间共同做出治疗决策。确定了关键变量,并基于五个变量提出了一种算法:出血表型、肌肉骨骼状况、治疗依从性、静脉通路和生活方式。还设想了一种以患者为中心的补充性偏好工具,旨在探索个体患者的优先事项、偏好和目标。希望所提出的算法和设想的患者偏好工具将有助于为每位患者选择一种在预防出血方面尽可能有效的治疗方法,同时也考虑到患者的期望和优先事项。

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